GM-1 ganglioside and motor recovery following human spinal cord injury.

Neurological deficit resulting from spinal cord injury varies widely in severity, ranging from transient abnormal reflexes to lifelong complete absence of motor and sensory function. Medical treatment to aid damaged neurons to recover function has been very limited; therapeutic efforts have focused primarily on initial stabilization of fractures, hemodynamic resuscitation, and then aggressive rehabilitation to enhance the full development of any remaining neuronal activity. Pharmacological treatment to improve restoration of neurological function may be possible, however, as indicated by many animal studies and a few clinical studies with a number of agents. A recent clinical trial of GM-1 ganglioside conducted in patients with spinal cord injuries showed that GM-1 ganglioside enhanced the recovery of neurological function 1 year after major spinal cord injury. In addition to GM-1 ganglioside treatment, these patients received aggressive medical and surgical treatment, as well as methylprednisolone. Neurological recovery was assessed with the Frankel scale and the American Spinal Injury Association (ASIA) motor scale. The findings show enhanced motor recovery compared with placebo in the lower extremities, but not in the upper extremities, over time. This corresponds to improved function of axons passing through the site of injury. Analysis of individual motor groups showed that neurological recovery in the GM-1 ganglioside-treated patients increased in initially paralyzed muscles, enabling them to regain useful motor function; paretic muscles were not found to be strengthened. The study provides the basis for larger studies of GM-1 ganglioside and methylprednisolone, which are currently under way.